Articles tagged with Gene Therapy
A recent study published in Immunity reveals that human T-cell receptor genes exhibit unexpectedly high variability among individuals, with each person having a unique set of gene variants. The researchers identified 175 new gene variants originating from Neanderthals, which are present in up to 20% of modern humans in Europe and Asia.
Researchers from Rice University have developed a new approach to control gene expression using proteolysis targeting chimeras (PROTACs). By reengineering the PROTAC molecular infrastructure, they demonstrated the ability to achieve chemically induced dimerization (CID), allowing for precise control over gene activation in specific loc...
Researchers evaluate an integrated NGS system, delivering accurate diagnoses in under 24 hours and expanding targeted treatments available to patients with myeloid neoplasms. The assay identified 80-92% of genetic variants, demonstrating promising results for accelerating precision therapies.
Researchers from HKUMed discovered that somatic deletion of AKTIP promotes luminal breast cancer development and resistance to endocrine therapy. Blocking the alternative escape pathway with a JAK2/STAT3 inhibitor can overcome this resistance, offering new therapeutic possibilities for patients with AKTIP gene deletion.
Scientists have identified genes that play key roles in the development of coronary artery disease (CAD), a leading cause of death worldwide. The study found notable differences in gene activity between males and females, as well as between cells that were multiplying and those that were not.
Researchers discover gene therapy ophNdi1 that boosts mitochondrial performance in retinal ganglion cells, potentially treating glaucoma and age-related macular degeneration. The therapy shows protective effects in three models of mitochondrial dysfunction.
A novel form of chemoimmunotherapy has been proven to be a promising treatment for canine cancer, extending the lives of terminally ill dogs. The therapy uses modified Mesenchymal Stem Cells carrying a potent 'kill-switch' that induces anti-cancer immunity and improves quality of life.
Researchers at Children's Hospital of Philadelphia have developed a custom-built application to automate the determination of engraftment after hematopoietic stem cell transplant. The tool has been shown to improve accuracy of reported engraftments, reducing errors in neutrophil and platelet engraftment reporting.
Researchers have discovered an anti-ageing gene that can rewind the heart's biological age by 10 years, offering a potential target for patients with heart failure. The study found that administering the healthy mutant gene to elderly patients with severe heart problems improved their cardiac function and rejuvenated their immune system.
Researchers at Massachusetts General Hospital created a new class of technologies called CRISPR-associated transposases (CASTs) to overcome diverse disease-causing mutations. The optimized approach improves product purity and genome-wide specificity, offering a potential solution for inserting entire genes into the genome.
Researchers found four genetic populations of the finless porpoise in Chinese waters and three in the Gulf of Thailand, with unique matriline lineages. The study suggests ongoing gene flow among populations, requiring careful evaluation of inter-population connectivity for conservation action.
Researchers at University of Texas M. D. Anderson Cancer Center develop a novel mRNA delivery system using extracellular vesicles, which can initiate collagen production in cells and has potential for other mRNA therapies
A new approach to gene therapy for inherited blindness uses lipid nanoparticles to deliver mRNA inside the eye, targeting light-sensitive cells and creating proteins that edit vision-harming gene mutations. The technology has shown promising results in animal studies, including mice and nonhuman primates.
A new gene therapy treatment has shown promising results in a clinical trial for Artemis-SCID patients, improving their immune function and reducing treatment complications. The treatment involves adding a healthy copy of the Artemis gene to the patient's own cells, resulting in improved T-cell and B-cell immunity.
The use of 3D-patient tumor avatars (3D-PTAs) is crucial for guiding treatment decisions in precision oncology. These avatars, including patient-derived organoids, 3D bioprinting, and microscale models, can accurately depict a tumor with its microenvironment, enabling the testing and prediction of therapeutic drug efficacy.
Scientists at Duke University have made a breakthrough in controlling gene expression in response to injury, using a segment of fish DNA called TREE. The method successfully targeted gene activity to specific regions and time windows, showing promise for regenerating damaged tissues in mammals.
Researchers from Japan have developed an RNA interference method using antisense oligonucleotides to correct a genetic defect in Fukuyama Muscular Dystrophy. This approach has shown promise in treating patients with the disease, which is characterized by generalized muscle weakness and intellectual disability.
A study by Sanford Burnham Prebys found that misfolded proteins in liver cells contribute to liver cancer development, potentially increasing the risk of certain patients receiving gene therapies for hemophilia. Improving FVIII folding could decrease this risk and develop safer treatments.
Researchers have discovered that a specific mutation in the misfolding protein causing Parkinson's disease can also protect against multiple system atrophy (MSA), another fatal neurodegenerative disorder. The findings provide a promising lead for developing targeted treatments using personalized medicine approaches.
Researchers have developed an AAV-based gene therapy to treat glutaric aciduria type I (GA-I), a rare genetic disorder causing accumulation of glutaric acid in the central nervous system. The treatment has shown promising results, with a survival rate of 100% in mice treated intracerebroventricularly.
Researchers at University of Pittsburgh have designed novel nanoparticles that co-deliver a chemotherapy drug and a novel immunotherapy, shrinking tumors in mouse models of colon and pancreatic cancer. The therapy silences a gene involved in immunosuppression by blocking Xkr8 protein distribution on the cell membrane.
Nucleic acid therapies aim to treat genetic disorders and diseases, but delivering therapeutics is a significant challenge. Researchers are investigating nanoparticle delivery systems to target specific cells and sub-cellular compartments for effective delivery.
Researchers have engineered a family of adeno-associated viral vectors that can deliver cargo to the primate brain, offering a safer and more efficient way to treat genetic diseases. The PAL family of AAVs has been shown to be three times better at delivering their cargo into the brain than current leading AAV delivery vehicle AAV9.
The UC San Diego Alpha Stem Cell Clinic will receive $8 million in funding to launch new clinical trials and improve accessibility of stem cell therapies. The clinic has already launched 59 clinical trials and treated 277 patients with various diseases.
The USC+CHLA Alpha Clinic will advance clinical trials of new cell and gene therapies, engaging underserved communities and training the workforce that conducts them. This program complements existing efforts to translate discoveries into new therapies for better health outcomes.
Researchers found that IGF1 gene therapy increases kisspeptin expression and GnRH release, and alters microglial cell numbers, suggesting a potential protective effect against reproductive decline. This could lead to new strategies for optimizing lifespan and combating age-related health problems in women.
A Rice University bioengineer has developed a noninvasive technology to measure gene expression in deep tissues, particularly in the brain. This innovation could improve the monitoring of gene therapy treating neurodegenerative disorders such as epilepsy, ALS, and Huntington's disease.
Researchers have developed a new gene therapy that selectively targets overactive brain cells, reducing excitability and suppressing seizures in mice. The treatment shows promise for treating neurological disorders such as epilepsy, Parkinson's disease, schizophrenia, and pain disorders.
Amanda Woerman's research aims to disrupt tau misfolding, a common thread in fatal neurodegenerative disorders PSP and CBD. The grant-funded study seeks to test a proof-of-concept gene therapy for these conditions, potentially leading to personalized medicine breakthroughs.
The Alpha Stem Cell Clinic will develop preclinical studies into early and later phase clinical trials with the goal of establishing advanced regenerative medicine treatments. The clinic will also foster greater collaboration with eight similar clinics across the state and educate the public about stem cell and related therapies.
Johns Hopkins Medicine researchers have developed a novel genetic engineering approach to deliver gene therapy by utilizing a cell's natural process to
A new Northwestern Medicine study identifies common and rare gene mutations that impact radiation resistance and sensitivity. This information will allow clinicians to better calibrate radiation doses based on genetic mutations, improving treatment efficacy while reducing toxicity.
Researchers at the University of Pennsylvania School of Medicine have developed a gene therapy that rapidly restores night vision in adults with LCA, a common form of congenital blindness. The treatment has been shown to improve rod-based visual functions and correct severe visual deficits in patients.
Nanoscope Technologies received a $1.5M NIH SBIR grant to develop gene-agnostic glaucoma therapy utilizing Engineered Mechanosensitive Channels (EMCs). EMCs can autonomously regulate pressure, offering a novel approach to treating Glaucoma.
Researchers at CNIO have identified alveolar type II pneumocytes as the primary cell type responsible for developing pulmonary fibrosis. The study reveals that targeting these cells through telomere-based therapy may lead to a breakthrough in treating this debilitating disease.
Researchers have used single-cell sequencing to uncover novel gene expression patterns in injured kidney cells, providing new avenues for biomarker discovery and treatment. The studies reveal that epithelial cells of all tubule segments are involved in the injury processes, with distinct molecular patterns across patients.
Researchers at Universidad de Navarra identified a biomarker that predicts CAR T cell therapeutic capacity, which could improve treatment outcomes for patients. The study found that high CAR density in CAR T cells is associated with a worse clinical response in hematological tumors.
Researchers developed a computational platform to identify metabolic vulnerabilities in ovarian cancer genes, suggesting opportunities for targeted therapies. The study found that certain genetic alterations can create vulnerabilities in cancer cell metabolism, which can be exploited to selectively kill cancer cells.
Researchers developed new educational materials to help individuals with sickle cell disease and their support networks learn about gene therapy clinical trials, benefits, risks, and treatment options. The project aims to facilitate engagement between the community and those conducting relevant clinical trials.
Scientists at St. Jude Children's Research Hospital have created a tool to identify 'safe harbor' sites for gene therapies, balancing safety and therapeutic expression. The tool uses genomic and epigenetic information to find regions with high variability among healthy individuals, reducing the risk of cancer or other problems.
Researchers discovered that autophagy facilitates the elimination of cancer cells via cell competition, highlighting its potential as a target for cancer prevention and treatment. The study sheds light on the role of autophagy in maintaining tissue homeostasis and opening avenues for novel anti-cancer therapeutics.
Researchers at UT Southwestern have identified a gene therapy target for polycystic kidney disease (PKD), which could lead to the arrest or cure of the condition. By blocking the inhibition of PKD1 and PKD2 gene expression, the researchers found that kidney cyst growth was hindered in ADPKD models.
New research estimates the overall disease burden of genetic risk factors on healthy life years lost, prioritizing interventions using genetic information. The study found that common genetic variants for cardio-vascular diseases and Alzheimer’s disease have a substantial population-level impact.
Researchers have developed a gene therapy that rescues cilia defects in retinal cells affected by Leber congenital amaurosis, a disease causing blindness in early childhood. The therapy restores opsin protein concentration in photoreceptor outer segments, suggesting functional NPHP5 may stabilize the primary cilium gate.
Breast cancer recurrence and metastatic spread remain a significant challenge, with researchers identifying a metabolic signature that can predict patient outcomes. The signature could be used to develop new therapies targeting cancer metabolism.
A novel stem cell-gene therapy has been shown to be safe in humans, with no serious side effects reported in the first trial. The treatment targets motor neurons that die in patients with amyotrophic lateral sclerosis (ALS), a fatal neurological disorder.
Researchers have developed a novel DNA-based method to identify people at greater genetic risk of developing Alzheimer's disease before symptoms appear. The method uses data on common DNA variants from over 7.1 million individuals and predicts a person's risk of Alzheimer's, depending on which DNA variants they have.
A new study identifies how the suppression of a specific transcription gene triggers changes that impair oligodendrocyte function in Huntington's disease. The researchers believe replacing or fixing defective glia cells may prove a far easier proposition than replenishing neurons lost in the disease.
A new preclinical model of thymic cancer reveals how a common mutation sparks tumor formation and identifies potential targets for therapy. The model, developed by Weill Cornell Medicine researchers, shares molecular characteristics with human thymic tumors, paving the way for new treatments.
Researchers from Trinity College Dublin have developed a new gene therapy approach targeting mitochondrial function to treat dry AMD, a debilitating eye disease. The therapy has shown benefit in multiple models of the condition, offering hope for a potential treatment that could help millions globally.
Researchers at UVA have discovered the mechanism behind gene regulation during organ development, shedding light on how genetic material interacts with transcription factors to create different cell types. The study's findings could offer insights into the initiation of certain cancers and inspire new therapeutic development.
A team of researchers has identified two compounds that target DNMT3A complexes in cancer cells, offering a safer and more targeted treatment for leukemia patients. The findings provide hope for reducing off-target toxicity associated with current leukemia therapies.
A new study has partly restored the function of retina's cone receptors in two completely colorblind children using gene therapy. The treatment has been shown to activate previously dormant communication pathways between the retina and brain, drawing on the plastic nature of the developing adolescent brain.
Researchers found that using the drug erlotinib, which blocks a specific cancer pathway, led to a 30 percent reduction in polyps formed in patients with FAP. The study's findings suggest potential ways to prevent colorectal cancer in the general population at an earlier stage.
Researchers found that administering tisagenlecleucel at the higher end of the approved dose range significantly improves overall and event-free survival in young patients. The study suggests that using higher doses of CAR-T therapy can achieve more effective and long-term responses without increasing toxicity risk.
A study published in Molecular Therapy — Methods & Clinical Development shows that delivering the protein EPS8 via gene therapy can rescue malfunctioning inner ear hair cells that transduce sound. In mice affected by recessive deafness, EPS8 increases stereocilia length and restores hair cell function.
A team of researchers from The Mount Sinai Hospital has made a groundbreaking discovery into the genetic and molecular mechanisms that predispose individuals to Alzheimer's disease. They identified 21 candidate risk genes, including SPI1, which regulates microglia and AD risk.
A new gene therapy approach using the neuroprotective protein SynCav1 has shown promising results in slowing down ALS disease progression and increasing life span in rodent models. The treatment preserved spinal cord motor neurons and extended longevity in mice, with similar effects observed in a rat model of ALS.
Researchers discover gene AVIL responsible for deadly brain tumor also causes two forms of childhood cancer, rhabdomyosarcoma. Blocking AVIL activity prevents formation of the disease in lab samples and mouse models.
Researchers used Guardant NGS to analyze nearly 17,000 lung cancer samples and found MET amplification in 1.2% of cases, with 20.8% having overlapping oncogenic drivers. The study suggests that high gene copy numbers and smaller amplified regions can be used to enrich for the true MET-sensitive population.